A functional genomics approach to dissect the molecular bases of CDKL5 Deficiency Disorder

Awardee: Davide Cacchiarelli, PhD

Institution: Telethon Institute of Genetics and Medicine

Award Amount: $150,000

Funding Period: May 1, 2022 - April 31, 2023

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Rescuing functional defects in a zebrafish model of CDKL5 deficiency: Contribution to the identification of therapeutic target

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Hematopoietic stem cell gene therapy as a therapeutic approach for CDKL5 Deficiency Disorder